ART002 / AccurEdit Therapeutics - LARVOL DELTA

Efficacy and Safety of ART002, a Single-dose Gene Editing Therapy, in Patients with Severe Heterozygous Familial Hypercholesterolemia (ASGCT 2025) - P=N/A | "While siRNA has improved drug convenience and adherence, the average LDL-C reduction of Inclisiran over 4 years is only 44% even with maximally tolerated statins. ART002 is the first single-course gene-editing therapy targeting PCSK9 that has been clinically proven to be safe and effective for the treatment of severe HeFH across all treated patients to date. This innovative therapy may potentially revolutionize the treatment landscape for hypercholesterolemia and ASCVD. Disease Focus of Abstract:Genetic Diseases"

Clinical • Atherosclerosis • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Heterozygous Familial Hypercholesterolemia • Metabolic Disorders • APOB • PCSK9